Quoin Pharmaceuticals Secures Key Regulatory Designations and Reports Clinical Progress
Quoin Pharmaceuticals has secured Orphan Drug Designations for QRX003 in the U.S. and Europe for Netherton Syndrome, while advancing regulatory pathways in Japan and Saudi Arabia. The company reported clinical progress including expansion of pediatric studies and potential for a Priority Review Voucher worth up to $200 million.
Quoin Pharmaceuticals Ltd. has secured Orphan Drug Designations for QRX003 for Netherton Syndrome in both the United States and Europe, while advancing proposed expedited regulatory approval pathways in Japan and Saudi Arabia. The company also reported continued clinical progress for QRX003 in Netherton Syndrome clinical studies, including data supporting longer term durable treatment effect and safety profile.
The pediatric Netherton Syndrome study has been expanded to 7 children now actively treated with QRX003, representing the largest cohort of this age group ever studied. Ongoing clinical data continues to support development of QRX003 as a treatment for Peeling Skin Syndrome, with the study being expanded to 6 subjects.
QRX003 was awarded Rare Pediatric Disease Designation by the U.S. Food and Drug Administration for Netherton Syndrome, which, if approved, could result in the receipt of a freely tradable Priority Review Voucher with a potential value to Quoin of $150-$200 million in non-dilutive cash. The U.S. Rare Pediatric Disease Priority Review Voucher program was extended by Congress through September 30, 2029 as part of the Give Kids a Chance Reauthorization Act.
On January 20, 2026, Quoin filed an application for Breakthrough Medicine Designation with the Saudi Food and Drug Authority for QRX003. If granted, the designation could enable accelerated regulatory review and availability in Saudi Arabia as early as the second half of 2026. On January 27, 2026, Quoin submitted an application to Japan's Ministry of Health, Labour and Welfare seeking Orphan Drug Designation for QRX003, with confirmation that QRX003 qualifies for both Orphan Drug Designation and Fast Track review in Japan.
The company's proprietary rapamycin topical platforms have achieved target loadings, with clinical studies planned in the second half of 2026. Quoin is planning to initiate clinical testing of QRX003 in Ichthyosis and SAM syndrome, potentially putting the product on track for approval for four rare genetic diseases where there are currently no approved treatments.
Quoin closed a private placement financing on October 10, 2025, raising up to $104.5 million in gross proceeds. The company's NETHERTON NOW awareness campaign has reached nearly 2 million video views and more than 24 million impressions globally since launch.